Health Technology Assessment for Molecular Diagnostics: Practices, Challenges, and Recommendations from the Medical Devices and Diagnostics Special Interest Group.

BACKGROUND: Health technology assessments (HTAs) are increasingly used to inform coverage, access, and utilization of medical technologies including molecular diagnostics (MDx). Although MDx are used to screen patients and inform disease management and treatment decisions, there is no uniform approach to their evaluation by HTA organizations. OBJECTIVES: The International Society for Pharmacoeconomics and Outcomes Research Devices and Diagnostics Special Interest Group reviewed diagnostic-specific HTA programs and identified elements representing common and best practices. METHODS: MDx-specific HTA programs in Europe, Australia, and North America were characterized by methodology, evaluation framework, and impact. Published MDx HTAs were reviewed, and five representative case studies of test evaluations were developed: United Kingdom (National Institute for Health and Care Excellence's Diagnostics Assessment Programme, epidermal growth factor receptor tyrosine kinase mutation), United States (Palmetto's Molecular Diagnostic Services Program, OncotypeDx prostate cancer test), Germany (Institute for Quality and Efficiency in Healthcare, human papillomavirus testing), Australia (Medical Services Advisory Committee, anaplastic lymphoma kinase testing for non-small cell lung cancer), and Canada (Canadian Agency for Drugs and Technologies in Health, Rapid Response: Non-invasive Prenatal Testing). RESULTS: Overall, the few HTA programs that have MDx-specific methods do not provide clear parameters of acceptability related to clinical and analytic performance, clinical utility, and economic impact. The case studies highlight similarities and differences in evaluation approaches across HTAs in the performance metrics used (analytic and clinical validity, clinical utility), evidence requirements, and how value is measured. Not all HTAs are directly linked to reimbursement outcomes. CONCLUSIONS: To improve MDx HTAs, organizations should provide greater transparency, better communication and collaboration between industry and HTA stakeholders, clearer links between HTA and funding decisions, explicit recognition of and rationale for differential approaches to laboratory-developed versus regulatory-approved test, and clear evidence requirements.

The Value of Companion Diagnostics: Overcoming Access Barriers to Transform Personalised Health Care into an Affordable Reality in Europe.

Personalised health care is an evolution, moving away from a disease-focused model of care, translating scientific and technological advances into benefits for patients, and placing them at the centre of the patients' health and care. Companion diagnostics emerge as a very specific and special group of in vitro diagnostics among the different technologies shaping the personalised health care spectrum. Companion diagnostics provide highly valuable information, allowing patients, health practitioners and payers to decide with a higher level of certainty on the potential benefits of a treatment or care pathway. Decreasing uncertainty may result in a more efficient selection of treatments and care, targeted at subpopulations that are most likely to benefit. Companion diagnostics account for a minimal portion of the already small expenditure on in vitro diagnostics (far less than 1% of total health care expenditure), and yet they provide the means to limit inefficient use of health care resources while optimising patient outcomes. It is clear that equal access to personalised health care is still an issue across the EU. One of the most common perceived barriers is affordability. The investment in companion diagnostics can provide long-term value for patients and health care systems, shifting resources to areas of need. Health systems do not fully recognise yet the value that companion diagnostics bring to make personalised health care more affordable across the EU. This inhibits patient access to personalised treatments and care, preventing improved outcomes. In many countries, market access frameworks for diagnostic tests are fragmented and not aligned with specific funding and reimbursement mechanisms, discouraging the use of these tests. Emerging evidence shows that patients are missing out on the appropriate tests and treatments while a reduction in the inefficient use of health care resources is not realised. This article outlines some of these market access barriers for companion diagnostics in the EU, including reimbursement challenges specific to some member states (Germany, the UK, and France). Furthermore, proposals addressing barriers and increasing timely patient access to companion diagnostics in the EU are presented.

Point-of-Care Testing for Clostridium Difficile Infection: A Real-World Feasibility Study of a Rapid Molecular Test in Two Hospital Settings.

INTRODUCTION: In the developed world, Clostridium difficile infection (CDI) is the most important cause of nosocomial infectious diarrhea. In addition to providing epidemiological data and helping to indicate that a local outbreak may be occurring, laboratory tests are used to augment clinical decisions on individual patients. Very rarely do diagnostic tests provide results at the point of decision making; in the intervening period between requesting investigations on a patient with suspected CDI and return of the laboratory result, decisions must be made regarding patient isolation and treatment. METHODS: A 22-month, real-world feasibility study was conducted in patients with clinically significant diarrhea, in a London Hospital between March 2011 and January 2013, in three older persons' wards and two intensive care units (ICUs) to determine acceptability, ease of use, change in turnaround time and clinical utility of a rapid, polymerase chain reaction (PCR)-based point-of-care test (POCT) (Cepheid GeneXpert(®), Sunnyvale, California, USA) for diagnosis of Clostridium difficile. Nurses in the older persons' ward and laboratory technicians in the ICU were trained to perform the test. Residual samples were sent to the centralized laboratory for parallel testing using a two-step algorithm. RESULTS: A total of 335 samples were tested using the POCT with a median turnaround time of 1.85 h compared with 18 h for the centralized laboratory test. Overall agreement with centralized laboratory testing was 98.1%. Discrepant samples were more frequent on elderly wards than ICU. Overall 20/335 (6%) processing errors were encountered and were highest in the first few months of the study. Significantly more processing errors occurred on the older persons' wards 13/102 (12.7%) than on ICU 7/271 (2.6%). Older persons' patients who had POCT were significantly less likely to have a test requested for bacterial stool culture (3.1% vs. 10.9% p = 0.044). This difference was not observed in the ICU patients. No other differences in ancillary test requesting, mortality or length of stay were observed. CONCLUSIONS: The majority of users reported that the POCT was easy to perform and was an acceptable part of their job. POCT using this system is feasible and acceptable to nursing staff and technicians working within these two hospital-based settings.